Phase 2
N=64
Safety and Tolerability Study of 2 Dose Level of Arikayce™ in Patients With Bronchiectasis and Chronic Infection Due to Pseudomonas Aeruginosa.
Bronchiectasis
Bottom Line
View on ClinicalTrials.gov: NCT00775138 ↗Enrolled (actual)
64
Serious AEs
4.8%
Results posted
Jul 2019
Primary outcome: Primary: Number of Participants Reporting Treatment-emergent AEs (TEAEs) up to End of Treatment — 2; 3; 2; 3 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- 280 mg Arikayce™ (Drug); Matching Placebo for Cohort 1 (Drug); 560 mg Arikayce™ (Drug); Matching Placebo for Cohort 2 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Insmed Incorporated
- Primary completion
- May 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Reporting Treatment-emergent AEs (TEAEs) up to End of Treatment |
2; 3; 2; 3; 3; 0 | — |
| PRIMARY Treatment-emergent Marked Laboratory Abnormalities up to 28 Days After Study Medication Discontinuation |
1; 0; 0; 0; 4; 0 | — |
| PRIMARY Treatment-emergent Pulmonary Function Test (PFT) for Acute Tolerability Assessment |
1.917; 1.841; 1.939; 1.758; 1.922; 1.820 | — |
| PRIMARY Treatment-emergent PFT Abnormalities up to the End of Study |
22; 10; 17; 9; 2; 0 | — |
| PRIMARY Number of Subjects With an Adverse Event Leading to Permanent Discontinuation of Study Medication |
0; 0; 1; 0 | — |
| PRIMARY Serious Adverse Events up to 28 Days After Study Medication Discontinuation |
1; 0; 1; 1 | — |
| SECONDARY Change From Baseline in Log10CFU Per Gram (Density) of Pseudomonas Aeruginosa in Sputum. |
-0.227; -0.333; -2.016; -0.474; -0.094; 0.315 | — |
| SECONDARY Total Pulmonary Symptom Severity Score (PSSS) |
-1.125; -0.200; -0.471; -0.250; -2.167; 0.000 | — |
| SECONDARY To Evaluate Change in St. George's Respiratory Questionnaire Measurements |
-4.024; -6.350; -6.101; -2.807; -6.205; -5.812 | — |
| SECONDARY To Evaluate the Use of Systemic Antipseudomonal Rescue Therapy |
0; 0; 0; 1; 0; 0 | — |
Summary
This is a study to determine the safety and tolerability of 28 days of daily dosing of two doses (280 mg and 560 mg) of Arikayce™ versus placebo in patients who have bronchiectasis and chronic infection due to Pseudomonas infection.
Eligibility Criteria
Inclusion Criteria
- Male or female study subjects≥ 18 years of age
- Confirmed diagnosis of multi-focal bronchiectasis in two or more lung segments by HRCT of the chest
- History of chronic infection with P. aeruginosa
- Confirmation of infection with P. aeruginosa at screening
- SaO2 ≥ 90% at Screening while breathing room air
- Ability to comply with study medication use, study visits, and study procedures as judged by the investigator
- Ability to produce at least 0.5 grams sputum or be willing to undergo an induction to produce sputum for clinical evaluation
Key Exclusion Criteria
- Forced Expiratory Volume in 1 second (FEV1) < 50% of predicted at Screening
- Patients with hemoptysis of ≥60 mL within 4 weeks prior to screening
- Bronchiectasis due to cystic fibrosis (CF), bronchopulmonary Aspergillus, aspiration of foreign body, or secondary to lung compression from tumors
- History of non-tuberculous mycobacterial and/or Aspergillus infection requiring treatment or treated within 2 years prior to screening
- Pulmonary tuberculosis requiring treatment or treated within two years prior to screening
- History of Lung transplantation
- Use of any inhalation or systemic antibiotics (IV antibiotics, or oral antibiotics) within 4 weeks prior to Study Day 1
- Evidence of biliary cirrhosis with portal hypertension
- Smoking tobacco or any substance within 6 months prior to screening, and throughout the study
- History of alcohol, medication, or illicit drug abuse within the 1 year prior to screening
Data sourced from ClinicalTrials.gov (NCT00775138). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.